Impact of long-term elosulfase alfa treatment on clinical and patient-reported outcomes in patients with mucopolysaccharidosis type IVA: results from a Managed Access Agreement in England

Table 1 MAA criteria for maintaining treatment

Clinical criteria Treatment-naïve patients: criteria following the first year of treatment • Improvement in 6MWT distance or the timed 25-foot (7.6 m) walk (T25FW) of ≥ 10% over baseline^a or stabilisation after 10% improvement^b • Improvement in FVC or FEV₁ of ≥ 5% over baseline or stabilisation after 1 year • Decline in LVEF of < 10% from baseline • Decline of uKS of ≥ 20% from baseline (and stabilised)
Ex-trial patients or patients receiving treatment for over 12 months • 6MWT or T25FW remains ≥ 5% above the baseline value at the start of treatment • FVC and FEV₁ remain ≥ 2% above the baseline value at the start of treatment • uKS levels remain reduced ≥ 20% from baseline • Decline in LVEF of < 10% from baseline
PRO criteria (same for treatment-naïve and ex-trial patients): • No adverse change in numerical value^c of two out of three of the following: • EQ-5D-5L score OR MPS-HAQ Caregiver Burden score • Beck Depression Score (≥ 13 years) • APPT/BPI pain severity score (depending on age)

6MWT 6-min walk test, APPT adolescent pediatric pain tool, BPI Brief Pain Inventory, EQ-5D-5L EuroQol 5 dimensions, 5 levels, FEV₁ forced expiratory volume in 1 s, FVC forced vital capacity, LVEF left ventricular ejection fraction, MPS-HAQ MPS Health Assessment Questionnaire, PRO patient-reported outcome, uKS urinary keratan sulphate
^aAs measured at the annual assessment
^b6MWT distance initially increased by 10% or more versus baseline, and then remained at least 5% above the baseline value at the start of treatment
^cThe MAA agreement did not specify how adverse change was defined for each instrument. Patients were assessed on a case-by-case basis where no adverse change means absolute scores remain stable (within the same category) or improve

ISSN: 1750-1172